Perinatal stem-cell and gene therapy for hemoglobinopathies

Seminars in Fetal & Neonatal Medicine
D V SurbekAnna M Wagner

Abstract

Most genetic diseases of the lymphohematopoietic system, including hemoglobinopathies, can now be diagnosed early in gestation. However, as yet, prenatal treatment is not available. Postnatal therapy by hematopoietic stem cell (HSC) transplantation from bone marrow, mobilized peripheral blood, or umbilical cord blood is possible for several of these diseases, in particular for the hemoglobinopathies, but is often limited by a lack of histocompatible donors, severe treatment-associated morbidity, and preexisting organ damage that developed before birth. In-utero transplantation of allogeneic HSC has been performed successfully in various animal models and recently in humans. However, the clinical success of this novel treatment is limited to diseases in which the fetus is affected by severe immunodeficiency. The lack of donor cell engraftment in nonimmunocompromised hosts is thought to be due to immunologic barriers, as well as to competitive fetal marrow population by host HSCs. Among the possible strategies to circumvent allogeneic HLA barriers, the use of gene therapy by genetically corrected autologous HSCs in the fetus is one of the most promising approaches. The recent development of strategies to overcome failure of effic...Continue Reading

References

Sep 1, 1995·Nature Medicine·C CoutelleU Froster
Feb 1, 1996·Nature Medicine·C A Blau, G Stamatoyannopoulos
Aug 20, 1996·Human Gene Therapy·J C Fletcher, G Richter
Dec 12, 1996·The New England Journal of Medicine·A W FlakeE D Zanjani
Feb 2, 1997·Bone Marrow Transplantation·B J BambachR J Jones
Apr 1, 1997·Ultrasound in Obstetrics & Gynecology : the Official Journal of the International Society of Ultrasound in Obstetrics and Gynecology·W Holzgreve
May 16, 1998·The Journal of Clinical Investigation·M C SenutF H Gage
Oct 15, 1998·Nature Medicine·J A AllayB P Sorrentino
Nov 25, 1998·British Journal of Haematology·C W TurnerW H Fleming
Mar 27, 1999·Human Gene Therapy·A WinklerR G Andrews
Jul 31, 1999·European Journal of Obstetrics, Gynecology, and Reproductive Biology·D V SurbekW Holzgreve
Sep 8, 1999·Science·B L ZieglerC Peschle
Oct 16, 1999·Science·E D Zanjani, W F Anderson
Nov 11, 1999·Proceedings of the National Academy of Sciences of the United States of America·G S LipshutzK M Gaensler
Feb 2, 2000·Nature Genetics·A L Caplan, J M Wilson
Feb 23, 2000·The Journal of Clinical Investigation·T H Bestor
Apr 28, 2000·Science·M Cavazzana-CalvoA Fischer
Jul 15, 2000·Ultrasound in Obstetrics & Gynecology : the Official Journal of the International Society of Ultrasound in Obstetrics and Gynecology·D V SurbekW Holzgreve
Feb 24, 2001·Human Reproduction Update·D V SurbekK H Nicolaides
Oct 22, 2002·American Journal of Obstetrics and Gynecology·Daniel V SurbekWolfgang Holzgreve
Jan 17, 2003·The New England Journal of Medicine·Salima Hacein-Bey-AbinaAlain Fischer
May 14, 2003·Stem Cells·Laurence E ShieldsRobert G Andrews
Oct 4, 2003·American Journal of Obstetrics and Gynecology·Alan J YoungDaniel V Surbek
Oct 7, 2004·American Journal of Obstetrics and Gynecology·Andreina SchoeberleinDaniel V Surbek

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Citations

Sep 17, 2009·Molecular Therapy : the Journal of the American Society of Gene Therapy·Nestor W MezaJose C Segovia
Aug 21, 2012·Best Practice & Research. Clinical Obstetrics & Gynaecology·Ruth Chadwick, Richardo Childs
May 23, 2014·Biochemical Society Transactions·Paolo De Coppi

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